BETHESDA, Md., April 08, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announces the appointment of Gene Mack, MBA as its chief financial officer, effective as of April 8, 2024. "We are thrilled to welcome Gene as our new CFO," commented Gain CEO Matthias Alder. "His experience as an operational CFO with a track record ...

DOCUMENTS INCORPORATED BY REFERENCE Portions of the definitive Proxy Statement ("Proxy Statement") of Gain Therapeutics, Inc. to be filed pursuant to Regulation 14A of the general rules and regulations under the Securities Exchange Act of 1934, as amended, for the 2024 annual meeting of stockholders to be held within 120 days after the end of the Registrant's 2023 fiscal year are incorporated by reference into Part III of this Form 10-K. Table of Contents Table of Contents UNITED STATES SECURITIES AND EXCHA ...

Exhibit 99.1 Gain Therapeutics Reports Financial Results for Year End 2023 and Provides Corporate Update BETHESDA, Md., March 26, 2024 – Gain Therapeutics, Inc. (Nasdaq: GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today reports financial results for the year ended December 31, 2023 and provides a corporate update. Corporate Highlights from Q4 2023 to Date "With the tremendous pro ...

BETHESDA, Md., March 15, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced participation in the upcoming Public Ventures Discovery Day on March 19, 2024 at Old Parkland in Dallas, TX. Public Ventures Discovery DayDate: Tuesday, March 19, 2024Presentation: 2:30 PM CSTLocation: Pecan Room, Old Parkland, 3819 Maple A ...

BETHESDA, Md., March 15, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announced participation in the upcoming Public Ventures Discovery Day on March 19, 2024 at Old Parkland in Dallas, TX. Public Ventures Discovery DayDate: Tuesday, March 19, 2024Presentation: 2:30 PM CSTLocation: Pecan Room, Old Parkland, 3819 Maple ...

BETHESDA, Md., March 05, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, today announces the presentation of a poster at the International Conference on Alzheimer's and Parkinson's Diseases and Related Neurological Disorders (AD/PD™ 2024) that addresses the mechanism of action of the Company's lead compound, GT-02287. AD/PD™ 20 ...

BETHESDA, Md., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule therapies, announces the initiation of the Multiple Ascending Dose (MAD) part of the Phase 1 clinical trial of GT-02287, Gain's lead drug candidate for the treatment of GBA1 Parkinson's disease. Based on the safety and tolerability profile observed in the Single Ascen ...

PART I - FINANCIAL INFORMATION [Item 1. Condensed Consolidated Financial Statements](index=5&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) The unaudited condensed consolidated financial statements for Gain Therapeutics, Inc. as of September 30, 2023, highlight decreased cash, increased net loss from higher operating expenses, and going concern uncertainty [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The balance sheet as of September 30, 2023, shows a significant decrease in total assets to $14.1 million from $24.1 million at year-end 2022, primarily due to a reduction in cash, cash equivalents, and marketable securities Condensed Consolidated Balance Sheet Highlights (Unaudited) | Metric | September 30, 2023 (USD) | December 31, 2022 (USD) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $4.3M | $7.3M | | Marketable securities (current & non-current) | $8.0M | $14.8M | | Total Assets | $14.1M | $24.1M | | **Liabilities & Equity** | | | | Total Liabilities | $6.8M | $5.2M | | Accumulated Deficit | $(56.1M) | $(38.5M) | | Total Stockholders' Equity | $7.3M | $18.9M | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) For the nine months ended September 30, 2023, the company reported a net loss of $17.5 million, a significant increase from the $12.9 million loss in the same period of 2022, primarily driven by increased R&D and G&A expenses Operating Results (Unaudited) | Metric | Nine Months Ended Sep 30, 2023 (USD) | Nine Months Ended Sep 30, 2022 (USD) | | :--- | :--- | :--- | | Total Revenues | $55K | $140K | | Research and Development Expenses | $(9.1M) | $(6.1M) | | General and Administrative Expenses | $(8.8M) | $(7.3M) | | Loss from Operations | $(17.8M) | $(13.2M) | | Net Loss | $(17.5M) | $(12.9M) | | Net Loss Per Share (basic and diluted) | $(1.42) | $(1.09) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the nine months ended September 30, 2023, net cash used in operating activities increased to $13.2 million, while investing activities provided $7.1 million and financing activities provided $3.1 million, resulting in a $3.0 million overall decrease in cash Cash Flow Summary (Unaudited) | Metric | Nine Months Ended Sep 30, 2023 (USD) | Nine Months Ended Sep 30, 2022 (USD) | | :--- | :--- | :--- | | Cash used in operating activities | $(13.2M) | $(10.8M) | | Cash provided by/(used in) investing activities | $7.1M | $(14.9M) | | Cash provided by/(used in) financing activities | $3.1M | $(57K) | | Net decrease in cash, cash equivalents and restricted cash | $(3.0M) | $(26.0M) | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail accounting policies and financial statement line items, critically disclosing a 'Going Concern' evaluation indicating insufficient cash to fund operations for 12 months and plans for additional capital - The company is a biotechnology firm developing small molecule therapeutics using its in-licensed SEE-Tx® platform for CNS disorders, LSDs, and oncology[37](index=37&type=chunk) - There is **substantial doubt** about the company's ability to continue as a going concern. Existing cash, cash equivalents, and marketable securities of **$12.3 million** are not sufficient to fund operations for at least 12 months from the report's issuance date. The company will need to raise additional capital to continue operations beyond Q3 2024[46](index=46&type=chunk)[47](index=47&type=chunk) - During the nine months ended September 30, 2023, the company sold **740,131 shares** under its ATM Program for gross proceeds of **$3.5 million**. As of September 30, 2023, **$12.5 million** remained available under the program[120](index=120&type=chunk)[50](index=50&type=chunk) - As of September 30, 2023, the company had research commitments of **$4.6 million** for activities to be performed within one year[142](index=142&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=29&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section analyzes the company's financial condition and operations, highlighting the initiation of a Phase 1 clinical trial for GT-02287, significant financial challenges including a going concern doubt, and plans for capital raising [Overview and Recent Developments](index=29&type=section&id=Overview%20and%20Recent%20Developments) The company, a biotechnology firm utilizing its SEE-Tx® platform, initiated a Phase 1 clinical trial for its lead candidate, GT-02287, in September 2023, while streamlining operations and seeking partnerships for capital efficiency - The company's lead product candidate, **GT-02287** for GBA1 Parkinson's disease, received approval to conduct a Phase 1 clinical trial in Australia in September 2023[145](index=145&type=chunk) - The Phase 1 trial for **GT-02287** is expected to be completed in the **first half of 2024**. The first cohort of the single ascending dose part was completed with no drug-related adverse effects observed[145](index=145&type=chunk) - In response to the current financing environment, the company is streamlining operations, pursuing partnering opportunities, and seeking non-dilutive funding to advance its pipeline[146](index=146&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) The company's net loss increased for both the three and nine-month periods ended September 30, 2023, primarily due to higher Research and Development and General and Administrative expenses, alongside a decrease in collaboration revenues Comparison of Operating Expenses (Nine Months Ended Sep 30) | Expense Category | 2023 (USD) | 2022 (USD) | Change | Reason for Change | | :--- | :--- | :--- | :--- | :--- | | Research & Development | $9.1M | $6.1M | +$3.0M | Increase in fees for third-party personnel assisting with the Parkinson's disease program | | General & Administrative | $8.7M | $7.2M | +$1.5M | Increase in legal fees (IP & corporate), accounting services, and personnel costs | - For the nine months ended September 30, 2023, total revenues were **$55 thousand**, down from **$140 thousand** in the same period of 2022, mainly from the Zentalis Collaboration Agreement[174](index=174&type=chunk) [Liquidity and Capital Resources](index=35&type=section&id=Liquidity%20and%20Capital%20Resources) As of September 30, 2023, the company had $12.3 million in cash, cash equivalents, and marketable securities, which are only sufficient to fund operations through Q3 2024, necessitating additional capital raising efforts - As of September 30, 2023, the company had **$12.3 million** in cash, cash equivalents, and marketable securities, with an accumulated deficit of **$56 million**[179](index=179&type=chunk) - The company's cash is expected to be sufficient to fund operations only through the **third quarter of 2024**, which raises substantial doubt about its ability to continue as a going concern[179](index=179&type=chunk) - The company has an ATM Program with **$12.5 million** of remaining capacity as of September 30, 2023, which it plans to use along with other financing options to raise capital[184](index=184&type=chunk)[186](index=186&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is a "smaller reporting company" and is therefore not required to provide the information for this item - As a "smaller reporting company," the registrant is not required to provide quantitative and qualitative disclosures about market risk[203](index=203&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of September 30, 2023, with no material changes in internal control over financial reporting during the quarter - Management concluded that as of September 30, 2023, the company's disclosure controls and procedures were **effective** at the reasonable assurance level[204](index=204&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, internal controls[205](index=205&type=chunk) PART II - OTHER INFORMATION [Item 1. Legal Proceedings](index=40&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings and is unaware of any pending or threatened legal actions that could materially adversely affect its business or financial condition - The company is not currently a party to any material legal proceedings[210](index=210&type=chunk) [Item 1A. Risk Factors](index=40&type=section&id=Item%201A.%20Risk%20Factors) Key risks include substantial doubt about the company's going concern ability, early-stage product development, reliance on third parties and intellectual property, intense competition, and stock price volatility - There is **substantial doubt** about the company's ability to continue as a going concern due to a history of operating losses and the need for future financing[214](index=214&type=chunk)[216](index=216&type=chunk) - The company's product candidates are novel and in early development; success in preclinical studies may not be indicative of results in later clinical trials[214](index=214&type=chunk) - The company relies on a license from Minoryx Therapeutics for its core **SEE-Tx® technology**, and termination of this agreement would have a material adverse effect on the business[220](index=220&type=chunk)[313](index=313&type=chunk) - The company will need to raise additional capital, which may not be available on favorable terms, or at all, and could lead to dilution or require relinquishing rights to technologies[215](index=215&type=chunk)[291](index=291&type=chunk) - Global and macroeconomic conditions, including economic instability, political conflicts (e.g., Ukraine, Hamas-Israel), and supply chain disruptions, could adversely affect operations and financial condition[220](index=220&type=chunk)[366](index=366&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=80&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities during the period and confirms no material change in the planned use of proceeds from its March 2021 IPO - There were no unregistered sales of equity securities in the period[457](index=457&type=chunk) - The company received net proceeds of **$40.5 million** from its IPO in March 2021. There has been no material change in the planned use of these proceeds[458](index=458&type=chunk)[459](index=459&type=chunk) [Item 3. Defaults Upon Senior Securities.](index=80&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) Not applicable - Not applicable[460](index=460&type=chunk) [Item 4. Mine Safety Disclosures](index=80&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) Not applicable - Not applicable[461](index=461&type=chunk) [Item 5. Other Information](index=80&type=section&id=Item%205.%20Other%20Information) Not applicable - Not applicable[462](index=462&type=chunk) [Item 6. Exhibits](index=81&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the quarterly report, including corporate governance documents, material contracts, and required certifications - Lists all exhibits filed with the Form 10-Q, including corporate governance documents, material contracts, and required certifications[464](index=464&type=chunk)

[PART I - FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20FINANCIAL%20INFORMATION) This section provides the unaudited condensed consolidated financial statements and management's analysis of financial condition and operations [Item 1. Condensed Consolidated Financial Statements](index=5&type=section&id=Item%201.%20Condensed%20Consolidated%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements, including balance sheets, statements of operations, and cash flows, for the period ended June 30, 2023 [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This table provides a snapshot of the company's financial position, detailing assets, liabilities, and equity at specific dates | Financial Metric | June 30, 2023 ($) | December 31, 2022 ($) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $6,319,925 | $7,311,611 | | Marketable securities (current & non-current) | $9,873,209 | $14,768,442 | | Total current assets | $17,308,448 | $21,091,296 | | Total assets | $18,260,874 | $24,099,387 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $5,588,164 | $4,125,251 | | Total liabilities | $7,168,040 | $5,219,873 | | Total stockholders' equity | $11,092,834 | $18,879,514 | [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) This table summarizes the company's revenues, expenses, and net loss over specified periods | Metric | Three Months Ended June 30, 2023 ($) | Three Months Ended June 30, 2022 ($) | Six Months Ended June 30, 2023 ($) | Six Months Ended June 30, 2022 ($) | | :--- | :--- | :--- | :--- | :--- | | Total revenues | $0 | $95,102 | $55,180 | $140,108 | | Research and development expenses | ($3,987,943) | ($2,582,224) | ($6,779,148) | ($4,138,664) | | General and administrative expenses | ($3,743,171) | ($2,689,263) | ($6,236,930) | ($4,466,306) | | Net loss | ($7,687,969) | ($5,085,420) | ($12,825,288) | ($8,358,063) | | Net loss per share - basic and diluted | ($0.62) | ($0.43) | ($1.05) | ($0.70) | [Condensed Consolidated Statements of Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) This table presents the net loss and other comprehensive income/loss components for the reporting periods | Metric | Three Months Ended June 30, 2023 ($) | Six Months Ended June 30, 2022 ($) | | :--- | :--- | :--- | | Net loss | ($7,687,969) | ($5,085,420) | | Comprehensive loss | ($7,649,956) | ($5,116,686) | [Condensed Consolidated Statement of Stockholders' Equity](index=8&type=section&id=Condensed%20Consolidated%20Statement%20of%20Stockholders%27%20Equity) This section details changes in the company's equity, including net loss and capital raises - Total stockholders' equity decreased from **$18.9 million** at December 31, 2022, to **$11.1 million** at June 30, 2023. The decrease was primarily driven by the net loss of **$12.8 million**, which was partially offset by **$3.0 million** in net proceeds from an at-the-market (ATM) stock offering[25](index=25&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This table outlines the cash inflows and outflows from operating, investing, and financing activities | Cash Flow Activity | Six Months Ended June 30, 2023 ($) | Six Months Ended June 30, 2022 ($) | | :--- | :--- | :--- | | Cash used in operating activities | ($9,238,779) | ($7,498,450) | | Cash provided by/(used in) investing activities | $5,152,086 | ($14,897,232) | | Cash provided by/(used in) financing activities | $2,946,272 | ($36,611) | | Net decrease in cash | ($990,688) | ($22,576,168) | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) These notes provide detailed explanations of the company's accounting policies, business operations, and financial statement items, including its going concern assessment - The company is a biotechnology firm developing small molecule therapeutics using its exclusively in-licensed computational platform, Site-Directed Enzyme Enhancement Therapy (SEE-Tx®), for CNS disorders, lysosomal storage disorders, metabolic disorders, and oncology[37](index=37&type=chunk) - Management believes the company has sufficient cash and cash equivalents to fund its operating expenses and capital expenditure requirements into the **third quarter of 2024** and for at least the next **twelve months** from the financial statement issuance date[49](index=49&type=chunk)[50](index=50&type=chunk) - During the six months ended June 30, 2023, the company sold **681,559 shares** of common stock under its At-The-Market (ATM) Program for aggregate gross proceeds of **$3.3 million**[121](index=121&type=chunk) - As of June 30, 2023, the company had research commitments of **$2.1 million** for activities that will be performed before the end of **Q2 2024**[140](index=140&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section discusses the company's financial condition, operational results, and future outlook, focusing on its therapeutic development and liquidity [Overview](index=28&type=section&id=Overview) This section provides a strategic overview of the company's business, product pipeline, and operational priorities - The company's lead product candidate, GT-02287 for GBA1 Parkinson's disease, has generated an extensive preclinical data package. An application to conduct a Phase 1 clinical trial in Australia was submitted in July 2023, with the study expected to begin in the **second half of 2023**[143](index=143&type=chunk) - The company is streamlining operational plans to improve capital efficiency and is pursuing partnering opportunities for its pipeline programs, including its lead Parkinson's program and lysosomal storage disease programs[145](index=145&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) This section analyzes the company's financial performance, detailing changes in revenues and expenses over time | Expense Category | Six Months Ended June 30, 2023 ($) | Six Months Ended June 30, 2022 ($) | Change ($) | | :--- | :--- | :--- | :--- | | Research and development | $6.8 million | $4.1 million | +$2.7 million | | General and administrative | $6.2 million | $4.5 million | +$1.7 million | - The increase in R&D expenses was primarily due to higher costs for third-party services related to the development of the company's Parkinson's disease program[172](index=172&type=chunk) - The increase in G&A expenses was mainly attributable to higher legal fees for intellectual property and corporate matters, professional accounting fees, and increased personnel-related costs[173](index=173&type=chunk) [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) This section assesses the company's ability to meet its short-term and long-term financial obligations - As of June 30, 2023, the company had cash, cash equivalents, and marketable securities totaling **$16.2 million**, down from **$22.1 million** at the end of 2022[176](index=176&type=chunk) - The company believes its existing cash resources will be sufficient to fund its operating expenses and capital expenditure requirements into the **third quarter of 2024**[178](index=178&type=chunk)[284](index=284&type=chunk) - The company has an At-The-Market (ATM) Program with approximately **$12.7 million** of remaining capacity as of June 30, 2023, to raise additional capital[180](index=180&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=37&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a "smaller reporting company," Gain Therapeutics is not required to provide the quantitative and qualitative disclosures about market risk - The company is a "smaller reporting company" and is not required to provide the information for this item[196](index=196&type=chunk) [Item 4. Controls and Procedures](index=37&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, evaluated the company's disclosure controls and procedures and concluded they were effective as of June 30, 2023. No material changes were made to the internal control over financial reporting during the quarter - Management, including the Chief Executive Officer and Chief Financial Officer, concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of June 30, 2023[197](index=197&type=chunk) - There were no changes in the company's internal control over financial reporting during the quarter that have materially affected, or are reasonably likely to materially affect, its internal control over financial reporting[198](index=198&type=chunk) [PART II - OTHER INFORMATION](index=38&type=section&id=PART%20II%20OTHER%20INFORMATION) This section covers legal proceedings, risk factors, equity sales, and other miscellaneous disclosures [Item 1. Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings and is not aware of any pending or threatened legal proceedings that could have a material adverse effect on its business - The company is not currently a party to any material legal proceedings[203](index=203&type=chunk) [Item 1A. Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant investment risks, including operating losses, capital needs, reliance on licensed technology, and regulatory challenges - The company has a history of operating losses and expects to incur losses for the foreseeable future, with no guarantee of ever achieving profitability[208](index=208&type=chunk) - The company will need to raise additional capital, which may not be available on favorable terms. Failure to do so could force delays or elimination of R&D programs[283](index=283&type=chunk)[284](index=284&type=chunk) - The business is significantly dependent on its exclusive license from Minoryx Therapeutics for the SEE-Tx® platform. Termination of this license would have a material adverse effect on the business[305](index=305&type=chunk) - The company faces risks from global macroeconomic conditions, including inflation, rising interest rates, and geopolitical instability, which could adversely affect its financial condition and operations[358](index=358&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=78&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section states there were no unregistered sales of equity securities during the period. It also confirms that the use of proceeds from the company's March 2021 IPO, which raised net proceeds of $40.5 million, has not materially changed from the plans described in the final prospectus - The company's IPO in March 2021 raised net proceeds of **$40.5 million**, and there has been no material change in the planned use of these proceeds[450](index=450&type=chunk)[451](index=451&type=chunk) [Item 3. Defaults Upon Senior Securities](index=78&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports that this item is not applicable - Not applicable[452](index=452&type=chunk) [Item 4. Mine Safety Disclosures](index=78&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) The company reports that this item is not applicable - Not applicable[453](index=453&type=chunk) [Item 5. Other Information](index=78&type=section&id=Item%205.%20Other%20Information) The company reports that this item is not applicable - Not applicable[454](index=454&type=chunk) [Item 6. Exhibits](index=79&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including corporate governance documents, material contracts, and certifications required by the Sarbanes-Oxley Act - Lists exhibits filed with the report, including certifications from the Principal Executive Officer and Principal Financial Officer pursuant to the Sarbanes-Oxley Act of 2002[456](index=456&type=chunk)

PART I—FINANCIAL INFORMATION Presents Gain Therapeutics' unaudited condensed consolidated financial statements and detailed notes for Q1 2023 and 2022 [Item 1. Condensed Consolidated Financial Statements](index=5&type=section&id=Item%201%20Condensed%20Consolidated%20Financial%20Statements) Presents unaudited consolidated financial statements and notes for Q1 2023 and 2022 [Condensed Consolidated Balance Sheets (Unaudited)](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20%28Unaudited%29) Details the company's financial position (assets, liabilities, equity) as of March 31, 2023, and December 31, 2022 Condensed Consolidated Balance Sheets (Unaudited) Table | Metric | Dec 31, 2022 ($) | Mar 31, 2023 ($) | | :--------------------------------- | :--------------- | :--------------- | | Cash and cash equivalents | 7,311,611 | 5,988,202 | | Marketable securities - current | 12,826,954 | 11,827,528 | | Total current assets | 21,091,296 | 19,321,984 | | Total assets | 24,099,387 | 21,322,575 | | Accounts payable | 1,626,100 | 2,213,489 | | Total current liabilities | 4,125,251 | 5,154,959 | | Total liabilities | 5,219,873 | 6,183,745 | | Total stockholders' equity | 18,879,514 | 15,138,830 | [Condensed Consolidated Statements of Operations (Unaudited)](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20%28Unaudited%29) Details the company's revenues, expenses, and net loss for the three months ended March 31, 2023, and 2022 Condensed Consolidated Statements of Operations (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Total revenues | 55,180 | 45,006 | | Research and development expenses | (2,791,205) | (1,556,440) | | General and administrative expenses | (2,493,759) | (1,777,043) | | Total operating expenses | (5,284,964) | (3,333,483) | | Loss from operations | (5,229,784) | (3,288,477) | | Net loss | (5,137,319) | (3,272,643) | | Net loss per share - basic and diluted | (0.43) | (0.28) | [Condensed Consolidated Statements of Comprehensive (Loss) (Unaudited)](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20%28Loss%29%20%28Unaudited%29) Reports net loss and other comprehensive income/loss for Q1 2023 and 2022 Condensed Consolidated Statements of Comprehensive (Loss) (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :---------------------------------- | :------------------------------ | :------------------------------ | | Net loss | (5,137,319) | (3,272,643) | | Unrealized gain on available-for-sale securities | 43,262 | — | | Foreign currency translation | 18,091 | (27,806) | | Other comprehensive income/(loss) | 60,683 | (23,657) | | Comprehensive loss | (5,076,636) | (3,296,300) | [Condensed Consolidated Statement of Stockholders' Equity (Unaudited)](index=11&type=section&id=Condensed%20Consolidated%20Statement%20of%20Stockholders%27%20Equity%20%28Unaudited%29) Outlines changes in stockholders' equity, including net loss and stock-based compensation Condensed Consolidated Statement of Stockholders' Equity (Unaudited) Table | Metric | Dec 31, 2022 ($) | Mar 31, 2023 ($) | | :--------------------------------- | :--------------- | :--------------- | | Balance as of period end | 18,879,514 | 15,138,830 | | Stock-based compensation | — | 565,432 | | Issuance of shares in ATM offering | — | 770,520 | | Net loss | (5,137,319) | (5,137,319) | [Condensed Consolidated Statements of Cash Flows (Unaudited)](index=13&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20%28Unaudited%29) Summarizes cash flows from operating, investing, and financing activities for Q1 2023 and 2022 Condensed Consolidated Statements of Cash Flows (Unaudited) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash used in operating activities | (4,217,415) | (2,447,391) | | Cash provided by/(used in) investing activities | 2,106,980 | (7,825) | | Cash provided by/(used in) financing activities | 748,908 | (37,974) | | Net (decrease)/increase in cash, cash equivalents and restricted cash | (1,323,105) | (2,542,507) | | Cash, cash equivalents and restricted cash at end of period | 6,019,324 | 34,369,445 | [Notes to Condensed Consolidated Financial Statements (Unaudited)](index=14&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements%20%28Unaudited%29) Provides detailed explanations of financial statement figures, business nature, and significant accounting policies [1. Nature of the Business and Basis of Presentation](index=14&type=section&id=1.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) Describes Gain Therapeutics' biotechnology focus, drug discovery platform, and ongoing financial challenges - Gain Therapeutics is a biotechnology company developing novel small molecule therapeutics for CNS disorders, LSDs, metabolic disorders, and oncology[36](index=36&type=chunk) - The company uses its exclusively in-licensed computational target and drug discovery platform, Site-Directed Enzyme Enhancement Therapy ("SEE-Tx®"), to discover novel allosteric binding sites and identify proprietary small molecules[36](index=36&type=chunk) - The company has incurred recurring losses and negative cash flows from operations since inception and anticipates needing additional capital to fund operations into the **third quarter of 2024**[43](index=43&type=chunk)[46](index=46&type=chunk) [2. Summary of Significant Accounting Policies](index=18&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) Outlines the key accounting principles and methods used in preparing the financial statements - The company's functional currency is USD, with foreign operations in Switzerland (Swiss Franc) and Spain (Euro), and translation adjustments are recorded in accumulated other comprehensive income/(loss)[51](index=51&type=chunk) - Marketable securities are classified as available-for-sale and carried at fair value, with unrealized gains/losses excluded from net income and accounted for in other comprehensive loss[55](index=55&type=chunk) - All costs incurred in performing research and development activities are expensed as incurred[88](index=88&type=chunk) [3. Cash, cash equivalents and restricted cash](index=29&type=section&id=3.%20Cash%2C%20cash%20equivalents%20and%20restricted%20cash) Details the composition of cash, cash equivalents, and restricted cash balances 3. Cash, cash equivalents and restricted cash Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------------- | :------------------- | :-------------------- | | Cash | 2,911,404 | 2,910,446 | | Money market | 3,076,798 | 4,401,165 | | Total cash and cash equivalents | 5,988,202 | 7,311,611 | | Restricted cash | 31,122 | 30,818 | - Restricted cash is held as a guarantee for expenses under the company's office lease agreement in Lugano[98](index=98&type=chunk) [4. Marketable Securities](index=30&type=section&id=4.%20Marketable%20Securities) Provides a breakdown of marketable securities, primarily U.S. Treasury Securities, and their fair values 4. Marketable Securities Table | Category | Amortized Cost ($) | Estimated Fair Value ($) | Gross Unrealized Gains ($) | Gross Unrealized Losses ($) | | :--------------------------------------- | :----------------- | :----------------------- | :----------------------- | :------------------------ | | Debt Securities - U.S. government treasury securities, current | 11,879,563 | 11,827,528 | — | (52,035) | | Debt Securities - U.S. government treasury securities, non-current | 987,370 | 988,388 | 1,018 | — | | Totals | 12,866,933 | 12,815,916 | 1,018 | (52,035) | - Marketable securities are primarily U.S. Treasury Securities with maturities from **April 2023 to April 2024**, classified as available-for-sale[99](index=99&type=chunk) [5. Prepaid Expenses and Other Current Assets](index=30&type=section&id=5.%20Prepaid%20Expenses%20and%20Other%20Current%20Assets) Itemizes the company's prepaid expenses and other current assets 5. Prepaid Expenses and Other Current Assets Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------------- | :------------------- | :-------------------- | | Tax credits | 137,383 | 103,877 | | Prepaid and deferred expenses | 730,108 | 552,882 | | Other receivables | 33,261 | 87,430 | | Prepaid D&O insurance costs | 605,502 | 208,542 | | Total | 1,368,871 | 848,854 | [6. Property and Equipment, net](index=32&type=section&id=6.%20Property%20and%20Equipment%2C%20net) Presents the net value of property and equipment after accumulated depreciation 6. Property and Equipment, net Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :----------------------- | :------------------- | :-------------------- | | Total property and equipment | 206,899 | 197,708 | | Less: accumulated depreciation | (62,263) | (53,329) | | Property and equipment, net | 144,636 | 144,379 | - Depreciation expense for the three months ended March 31, 2023, was **$8,300**, up from **$5,300** in the same period of 2022[103](index=103&type=chunk) [7. Operating Lease; Right of Use ("ROU") Assets](index=32&type=section&id=7.%20Operating%20Lease%3B%20Right%20of%20Use%20%28%22ROU%22%29%20Assets) Details the company's operating lease assets and liabilities, including lease terms and discount rates 7. Operating Lease; Right of Use ("ROU") Assets Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :------------------------------ | :------------------- | :-------------------- | | Operating lease- right of use assets | 609,877 | 659,933 | | Operating lease liability - current | 232,507 | 229,080 | | Operating lease liability - non-current | 385,922 | 441,784 | | Weighted average remaining lease term - years | 2.90 | 3.05 | | Weighted average discount rate | 1.52 | 1.53 | - Operating lease costs for the three months ended March 31, 2023, were **$60,601**, compared to **$58,170** in the prior year[106](index=106&type=chunk) [8. Accounts Payable](index=33&type=section&id=8.%20Accounts%20Payable) Reports the company's accounts payable balance as of the reporting dates - Accounts payable increased to **$2.2 million** as of **March 31, 2023**, from **$1.6 million** as of December 31, 2022[108](index=108&type=chunk) [9. Other Current Liabilities and Deferred Income](index=33&type=section&id=9.%20Other%20Current%20Liabilities%20and%20Deferred%20Income) Itemizes other current liabilities and deferred income balances 9. Other Current Liabilities and Deferred Income Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :------------------------------------ | :------------------- | :-------------------- | | Payable for social security and withholding taxes | 225,772 | 256,798 | | Accrued payroll | 990,025 | 660,556 | | Accrued expenses | 1,263,183 | 1,082,091 | | Tax provision | 120,783 | 107,311 | | Total other current liabilities | 2,599,763 | 2,106,756 | | Deferred income | — | 55,180 | [10. Pension Obligations](index=34&type=section&id=10.%20Pension%20Obligations) Details the company's funded status and costs related to pension obligations 10. Pension Obligations Table | Metric | March 31, 2023 ($) | December 31, 2022 ($) | | :--------------------------------- | :------------------- | :-------------------- | | Funded status at end of period | (164,568) | (157,580) | | Service cost (3 months ended Mar 31) | 35,078 | 169,709 | | Interest cost (3 months ended Mar 31) | 4,675 | 3,376 | [11. Loans](index=34&type=section&id=11.%20Loans) Describes the company's zero-interest loan and its future payment schedule - The company has a **CHF 638,000** (**$700,221** historical FX rate) **nine-year**, zero-interest loan obtained in August 2020, with quarterly payments of **CHF 20,000** starting December 31, 2021, and ending September 30, 2029[113](index=113&type=chunk) 11. Loans Table | Year | Loan Payments ($) | | :--------- | :---------------- | | Total | 587,496 | | 2024 | 109,200 | | 2025 | 87,360 | | 2026 | 87,360 | | 2027 | 87,360 | | 2028 | 87,360 | | Thereafter | 128,856 | [12. Fair Value Measurement](index=34&type=section&id=12.%20Fair%20Value%20Measurement) Presents financial assets measured at fair value, categorized by input levels 12. Fair Value Measurement Table | Category | March 31, 2023 ($) | December 31, 2022 ($) | | :--------------------------------------- | :------------------- | :-------------------- | | Marketable securities available for sale | 12,815,916 | 14,768,442 | | Cash equivalents: Money market funds | 3,076,798 | 4,401,165 | | Total financial assets | 15,892,714 | 19,169,607 | - All financial assets measured at fair value are classified as **Level 1** inputs, based on quoted prices in active markets[116](index=116&type=chunk)[119](index=119&type=chunk) [13. Common and Preferred Stock](index=36&type=section&id=13.%20Common%20and%20Preferred%20Stock) Details the number of common shares issued and outstanding, and recent ATM program activities - As of March 31, 2023, **12,087,142 shares** of common stock were issued and outstanding, compared to **11,883,368 shares** as of December 31, 2022[120](index=120&type=chunk) - During the three months ended March 31, 2023, the company sold **203,774 shares** of common stock under its ATM Program at an average price of **$4.48 per share**, generating aggregate net proceeds of **$0.8 million**[121](index=121&type=chunk)[122](index=122&type=chunk) [14. Equity Incentive Plans](index=38&type=section&id=14.%20Equity%20Incentive%20Plans) Outlines stock option, restricted stock unit, and performance restricted stock unit plans [Stock Option Grants](index=38&type=section&id=Stock%20Option%20Grants) Provides details on stock options outstanding, their fair value, and exercise prices Stock Option Grants Table | Metric | Dec 31, 2022 | Mar 31, 2023 | | :--------------------------------- | :----------- | :----------- | | Options outstanding | 1,879,662 | 2,438,512 | | Weighted Average Grant Date Fair Value | $2.94 | $3.05 | | Weighted Average Exercise Price | $4.42 | $4.50 | - **558,850 options** were granted during the three months ended March 31, 2023, with a weighted average grant date fair value of **$3.46**[128](index=128&type=chunk)[131](index=131&type=chunk) [Restricted Stock Units and Performance Restricted Stock Units](index=39&type=section&id=Restricted%20Stock%20Units%20and%20Performance%20Restricted%20Stock%20Units) Details outstanding restricted stock units, their fair value, and vesting periods Restricted Stock Units and Performance Restricted Stock Units Table | Metric | Dec 31, 2022 | Mar 31, 2023 | | :--------------------------------- | :----------- | :----------- | | RSUs outstanding | 103,050 | 295,550 | | Weighted average Grant Date Fair Value | $3.48 | $3.97 | | Weighted average Duration of Vesting Period (years) | 2.40 | 3.44 | - **192,500 RSUs** were granted during the three months ended March 31, 2023, with a weighted-average grant date fair value of **$4.24**[133](index=133&type=chunk) - Total unrecognized compensation cost related to non-vested stock options and RSUs was **$5.7 million** as of March 31, 2023, expected to be recognized over **4 years**[135](index=135&type=chunk) [15. Warrants](index=40&type=section&id=15.%20Warrants) Describes the outstanding equity-classified warrants, including exercise prices and expiration dates - **237,249** equity-classified warrants are outstanding with an exercise price of **$5.07 per share**, exercisable until **July 20, 2025**[136](index=136&type=chunk) - An additional **200,000** equity-classified warrants are outstanding with an exercise price of **$13.75 per share**, exercisable for **four years** from May 6, 2021[137](index=137&type=chunk) [16. Collaboration Agreement](index=40&type=section&id=16.%20Collaboration%20Agreement) Discusses the concluded multi-target collaboration agreement with Zentalis for cancer treatment - The multi-target collaboration agreement with Zentalis to discover new product candidates for cancer treatment has concluded[138](index=138&type=chunk)[141](index=141&type=chunk) [17. Net loss per common share](index=42&type=section&id=17.%20Net%20loss%20per%20common%20share) Presents basic and diluted net loss per common share and weighted-average shares outstanding 17. Net loss per common share Table | Metric | 3 Months Ended Mar 31, 2023 | 3 Months Ended Mar 31, 2022 | | :--------------------------------------- | :-------------------------- | :-------------------------- | | Net loss per share - basic and diluted | (0.43) | (0.28) | | Weighted-average common shares - basic and diluted | 11,935,081 | 11,883,368 | - Potentially dilutive securities (stock options, RSUs, PRSUs, and warrants) were excluded from diluted EPS calculation as their effect would be anti-dilutive due to the company's net losses[94](index=94&type=chunk)[142](index=142&type=chunk)[143](index=143&type=chunk) [18. Related Parties](index=42&type=section&id=18.%20Related%20Parties) Identifies related parties and outlines the exclusive license agreement with Minoryx Therapeutics SL - Minoryx Therapeutics SL is a related party due to shared board chairmanship[144](index=144&type=chunk) - The company has an exclusive worldwide license agreement with Minoryx for intellectual property, with royalty payments of **8%** on net revenues from products infringing composition of matter claims or Minoryx molecules, and **3%** on net revenues from products infringing method claims or Minoryx know-how[144](index=144&type=chunk)[147](index=147&type=chunk) [19. Commitments](index=42&type=section&id=19.%20Commitments) Details the company's research commitments for the current fiscal year - As of March 31, 2023, the company had **$3.2 million** in research commitments for activities to be performed in **fiscal year 2023**[146](index=146&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations.](index=44&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations.) Analyzes Gain Therapeutics' business, financial condition, and operating results for Q1 2023 vs Q1 2022 [Overview](index=44&type=section&id=Overview) Provides a high-level description of Gain Therapeutics' biotechnology focus and strategic operational plans - Gain Therapeutics is a biotechnology company developing novel small molecule therapeutics for CNS disorders, lysosomal storage disorders (LSDs), metabolic disorders, and oncology using its SEE-Tx® platform[150](index=150&type=chunk) - The lead product candidate, GT-02287 for GBA1 Parkinson's disease, has completed IND-enabling toxicology studies, with a **Phase 1 clinical trial** expected to begin in Australia in the **second half of 2023**[151](index=151&type=chunk) - The company is streamlining operational plans to become more capital efficient, focusing on Parkinson's and Gaucher disease programs, and seeking non-dilutive funding for other research programs, including Krabbe disease and alpha-1 antitrypsin deficiency, and new oncology programs[153](index=153&type=chunk)[154](index=154&type=chunk) [Recent Developments](index=46&type=section&id=Recent%20Developments) Highlights recent grants awarded and key personnel appointments - Awarded a **€1.2 million** (approx. **$1.3 million**) Eurostars and Innosuisse grant for the alpha-1 antitrypsin deficiency program, with research starting in **Q2 2023**[156](index=156&type=chunk) - Awarded a **CHF 2.5 million** (approx. **$2.8 million**) Innosuisse grant for the GBA1 Parkinson's disease program, with research starting in **Q2 2023**[157](index=157&type=chunk) - Appointed C. Evan Ballantyne as Chief Financial Officer on **May 3, 2023**[157](index=157&type=chunk) [Financial Condition](index=46&type=section&id=Financial%20Condition) Assesses the company's financial health, including accumulated deficit and capital runway - The company has incurred recurring losses and negative cash flows from operations since inception, with an accumulated deficit of **$43.6 million** as of **March 31, 2023** (vs. **$38.5 million** at Dec 31, 2022)[160](index=160&type=chunk) - As of **March 31, 2023**, cash, cash equivalents, and marketable securities totaled **$18.8 million**[160](index=160&type=chunk) - Management believes existing capital will fund operating expenses and capital expenditure requirements into the **third quarter of 2024**, but substantial additional financing is needed for product development and potential commercialization[161](index=161&type=chunk) [Financing Requirements; Current Financing Environment](index=48&type=section&id=Financing%20Requirements%3B%20Current%20Financing%20Environment) Discusses future capital needs and macroeconomic impacts on financing - Future cash needs are expected to be financed through public/private equity offerings (including ATM Program), debt financings, government/private grants, collaborations, strategic alliances, and licensing arrangements[162](index=162&type=chunk) - Worsening global macroeconomic conditions (war in Ukraine, geopolitical tension, inflation, rising interest rates, bank liquidity concerns, capital market volatility) may impact the ability to obtain additional financing on favorable terms[163](index=163&type=chunk) [Strategic Transactions; Collaboration and Licensing Agreement](index=48&type=section&id=Strategic%20Transactions%3B%20Collaboration%20and%20Licensing%20Agreement) Explains the company's approach to collaborations and licensing for its SEE-Tx® platform - The company enters into collaboration and licensing arrangements to use its SEE-Tx® computational platform technology to discover novel allosteric sites and identify proprietary small molecules[164](index=164&type=chunk) [Components of Our Consolidated Results of Operations](index=48&type=section&id=Components%20of%20Our%20Consolidated%20Results%20of%20Operations) Details primary components of consolidated results: limited revenue and significant R&D and G&A expenses [Revenue](index=48&type=section&id=Revenue) Discusses the company's historical and expected revenue sources - The company has not generated any revenue from product sales and does not expect to in the foreseeable future[165](index=165&type=chunk) - Historically, limited collaboration revenue was derived from the Zentalis collaboration agreement[165](index=165&type=chunk) [Total Operating Expenses](index=48&type=section&id=Total%20Operating%20Expenses) Explains the composition of the company's operating expenses - Operating expenses since inception have consisted solely of research and development and general and administrative costs[166](index=166&type=chunk) [Research and Development Expenses](index=48&type=section&id=Research%20and%20Development%20Expenses) Analyzes the trends and drivers of research and development expenditures Research and Development Expenses Table | Category | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Preclinical activities and outside services | 1,676,704 | 794,296 | | Personnel expenses | 980,576 | 726,954 | | Other | 133,925 | 69,899 | | Research grants | — | (34,709) | | Total research and development expenses | 2,791,205 | 1,556,440 | - The increase in R&D expenses was primarily due to increased third-party services related to Parkinson's and Gaucher disease programs[179](index=179&type=chunk) - R&D expenses are anticipated to increase substantially in future periods due to increased headcount, expanded infrastructure, increased insurance costs, and the commencement of clinical trials[169](index=169&type=chunk)[171](index=171&type=chunk) [General and Administrative Expenses](index=50&type=section&id=General%20and%20Administrative%20Expenses) Examines the changes and factors influencing general and administrative costs - General and administrative expenses increased by **$0.7 million** to **$2.5 million** for the three months ended March 31, 2023, compared to **$1.8 million** for the same period in 2022[180](index=180&type=chunk) - The increase was primarily due to higher legal fees (intellectual property and corporate matters), directors and officers insurance, professional accounting fees, and increased personnel-related costs (headcount and stock-based compensation)[180](index=180&type=chunk) [Other Financial Income (Expense)](index=52&type=section&id=Other%20Financial%20Income%20%28Expense%29) Presents non-operating financial income and expenses, including interest and foreign exchange Other Financial Income (Expense) Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Interest income, net | 152,035 | (1,651) | | Foreign exchange gain/(loss), net | (42,842) | 19,162 | [Consolidated Results of Operations](index=52&type=section&id=Consolidated%20Results%20of%20Operations) Summarizes net loss and key drivers for Q1 2023 compared to 2022 [Comparison of the Three Months ended March 31, 2023 and 2022](index=52&type=section&id=Comparison%20of%20the%20Three%20Months%20ended%20March%2031%2C%202023%20and%202022) Provides a detailed comparative analysis of financial performance between the two periods [Revenues](index=52&type=section&id=Revenues) Compares total revenues for the three months ended March 31, 2023, and 2022 Revenues Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :----------------------- | :------------------------------ | :------------------------------ | | Total revenues | 55,180 | 45,006 | [Research and Development Expenses](index=54&type=section&id=Research%20and%20Development%20Expenses) Compares research and development expenses for the three months ended March 31, 2023, and 2022 Research and Development Expenses Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Research and development expenses | 2,791,205 | 1,556,440 | [General and Administrative Expenses](index=54&type=section&id=General%20and%20Administrative%20Expenses) Compares general and administrative expenses for the three months ended March 31, 2023, and 2022 General and Administrative Expenses Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | General and administrative expenses | 2,493,759 | 1,777,043 | [Liquidity and Capital Resources](index=54&type=section&id=Liquidity%20and%20Capital%20Resources) Assesses the company's ability to meet short-term and long-term financial obligations - As of **March 31, 2023**, cash, cash equivalents, and marketable securities totaled **$18.8 million** (down from **$22.1 million** at Dec 31, 2022)[182](index=182&type=chunk) - The company had an accumulated deficit of **$43.6 million** as of **March 31, 2023**[182](index=182&type=chunk) - Management believes existing capital will fund operating expenses and capital expenditure requirements into the **third quarter of 2024**[182](index=182&type=chunk) - The company has approximately **$15 million** of remaining capacity under its ATM Program as of **March 31, 2023**[184](index=184&type=chunk) [Cash Flows](index=56&type=section&id=Cash%20Flows) Analyzes cash flows from operating, investing, and financing activities for Q1 2023 and 2022 [Cash Flows from Operating Activities](index=56&type=section&id=Cash%20Flows%20from%20Operating%20Activities) Details cash used in operating activities for the three months ended March 31, 2023, and 2022 Cash Flows from Operating Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------- | :------------------------------ | :------------------------------ | | Cash used in operating activities | (4,217,415) | (2,447,391) | [Cash Flows from Investing Activities](index=56&type=section&id=Cash%20Flows%20from%20Investing%20Activities) Details cash from investing activities for Q1 2023 and 2022 Cash Flows from Investing Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash provided by/(used in) investing activities | 2,106,980 | (7,825) | - Investing activities were positively impacted by **$4.1 million** from marketable securities maturities, partially offset by **$2.0 million** in purchases[190](index=190&type=chunk) [Cash Flows from Financing Activities](index=56&type=section&id=Cash%20Flows%20from%20Financing%20Activities) Details cash from financing activities for Q1 2023 and 2022 Cash Flows from Financing Activities Table | Metric | 3 Months Ended Mar 31, 2023 ($) | 3 Months Ended Mar 31, 2022 ($) | | :--------------------------------------- | :------------------------------ | :------------------------------ | | Cash provided by/(used in) financing activities | 748,908 | (37,974) | | Net (decrease)/increase in cash, cash equivalents and restricted cash | (1,323,105) | (2,542,507) | | Cash, cash equivalents and restricted cash at end of period | 6,019,324 | 34,369,445 | - Cash provided by financing activities was mainly related to the ATM Program[191](index=191&type=chunk) [Funding Requirements](index=57&type=section&id=Funding%20Requirements) Discusses the factors influencing future funding needs and potential financing strategies - Future funding requirements depend on the scope, timing, progress, and results of R&D, preclinical studies, and clinical trials for product candidates[193](index=193&type=chunk)[194](index=194&type=chunk) - The company expects to finance operations through equity offerings, debt financings, government/private grants, collaborations, strategic alliances, and licensing arrangements[194](index=194&type=chunk) - Inability to obtain additional funds on acceptable terms could lead to delays, reductions, or elimination of R&D programs, commercialization efforts, or curtailment of operations[194](index=194&type=chunk) [Critical Accounting Policies and Use of Estimates](index=58&type=section&id=Critical%20Accounting%20Policies%20and%20Use%20of%20Estimates) Highlights key accounting policies and estimates requiring significant management judgment - The preparation of financial statements requires estimates and judgments related to accrued expenses, defined benefit pension liability, share-based compensation, and research grants[195](index=195&type=chunk) - No material changes to critical accounting policies occurred during the three months ended March 31, 2023[195](index=195&type=chunk) [JOBS Act](index=58&type=section&id=JOBS%20Act) Explains the company's status as an "emerging growth company" and associated reporting exemptions - The company qualifies as an **"emerging growth company"** under the JOBS Act, allowing reliance on reduced reporting requirements[196](index=196&type=chunk)[419](index=419&type=chunk) - Exemptions include reporting only **two years** of audited financial statements, exemption from auditor attestation for internal control over financial reporting, and reduced executive compensation disclosures[196](index=196&type=chunk)[419](index=419&type=chunk) - The **"emerging growth company"** status will continue until the earliest of **December 31, 2026**, or if annual gross revenue exceeds **$1.07 billion**, or if **$1.0 billion** in non-convertible debt is issued, or if market value of non-affiliate held common stock exceeds **$700 million**[197](index=197&type=chunk)[420](index=420&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=58&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) States that as a "smaller reporting company," Gain Therapeutics is exempt from market risk disclosures - As a **"smaller reporting company,"** Gain Therapeutics is not required to provide quantitative and qualitative disclosures about market risk[198](index=198&type=chunk) [Item 4. Controls and Procedures](index=58&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective, with no material changes in internal control [Evaluation of Disclosure Controls and Procedures](index=58&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Management's assessment of the effectiveness of disclosure controls and procedures - Disclosure controls and procedures were evaluated by management, including the CEO and CFO, and concluded to be effective at the reasonable assurance level as of **March 31, 2023**[201](index=201&type=chunk) [Changes in Internal Control over Financial Reporting](index=60&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) Reports on any material changes in internal control over financial reporting during the period - No material changes in internal control over financial reporting occurred during the period covered by this Quarterly Report[202](index=202&type=chunk) [Inherent Limitations on Effectiveness of Controls](index=60&type=section&id=Inherent%20Limitations%20on%20Effectiveness%20of%20Controls) Acknowledges the inherent limitations of control systems in providing absolute assurance - Control systems provide reasonable assurance but cannot guarantee absolute assurance that objectives will be met or that all misstatements/fraud will be detected[203](index=203&type=chunk) PART II—OTHER INFORMATION Presents additional information not covered in financial statements, including legal, risk, and equity details [Item 1. Legal Proceedings.](index=60&type=section&id=Item%201.%20Legal%20Proceedings.) The company is not involved in material legal proceedings, nor aware of any threatened actions - The company is not currently a party to any material legal proceedings[205](index=205&type=chunk) - No pending or threatened legal proceedings are known that could adversely affect the business, operating results, or financial condition[205](index=205&type=chunk) [Item 1A. Risk Factors.](index=60&type=page&id=Item%201A.%20Risk%20Factors.) Details significant risks impacting operations, financial condition, and stock price [Risk Factor Summary](index=60&type=section&id=Risk%20Factor%20Summary) Provides a concise overview of the company's most significant operational and financial risks - The company has a history of operating losses and expects to incur losses for the foreseeable future, with no guarantee of generating revenues or achieving profitability[208](index=208&type=chunk)[211](index=211&type=chunk) - Significant risks include the inability to initiate or complete preclinical/clinical trials, challenges in patient enrollment for low-prevalence disorders, and the uncertainty of developing novel product candidates[213](index=213&type=chunk) - The company will need to raise additional capital, which may dilute stockholders, restrict operations, or require relinquishing rights to technologies/product candidates[213](index=213&type=chunk) - Other key risks include extensive government regulation, potential lack of market acceptance for approved products, reliance on licensed intellectual property (SEE-Tx® platform), and the adverse effects of global macroeconomic conditions and data privacy/security laws[213](index=213&type=chunk) [Risks Related to Our Business](index=62&type=section&id=Risks%20Related%20to%20Our%20Business) Discusses risks inherent to the business model, including operating losses and future success uncertainty - The company has a history of operating losses and expects to incur losses for the foreseeable future, with no significant revenues generated to date[211](index=211&type=chunk) - Future success is uncertain due to dependence on product candidate development, ability to scale operations, establish brand, expand to meet commercial demand, and minimize security risks[212](index=212&type=chunk) - Operating results are expected to fluctuate due to factors such as funding, preclinical/clinical trial progress, regulatory approvals, market acceptance, competition, and global macroeconomic conditions[217](index=217&type=chunk)[222](index=222&type=chunk) [Risks Related to Product Development, Regulatory Approval, Manufacturing and Commercialization](index=66&type=section&id=Risks%20Related%20to%20Product%20Development%2C%20Regulatory%20Approval%2C%20Manufacturing%20and%20Commercialization) Outlines risks in developing, approving, manufacturing, and commercializing product candidates - Conducting clinical trials outside the U.S. (e.g., Australia for Parkinson's program) carries risks, as the FDA may not accept foreign data, potentially delaying or halting development[221](index=221&type=chunk)[224](index=224&type=chunk) - Low prevalence of target disorders may lead to difficulties and delays in patient identification and enrollment for clinical trials, increasing development costs[233](index=233&type=chunk)[234](index=234&type=chunk) - Product candidates are novel and in early development, requiring extensive testing; success in early preclinical studies is not indicative of later clinical trial results, and there's a high failure rate in late-stage trials[239](index=239&type=chunk)[241](index=241&type=chunk)[243](index=243&type=chunk) - Product candidates may cause serious adverse events (SAEs) or undesirable side effects, which could delay or prevent marketing approval, or lead to market withdrawal or sales limitations[269](index=269&type=chunk)[270](index=270&type=chunk) [Risks Related to Our Financial Condition and Capital Requirements; Competition](index=83&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements%3B%20Competition) Addresses risks concerning financial stability, capital needs, and industry competition - Substantial additional capital is required for R&D, preclinical/clinical trials, and marketing, which may dilute stockholders, restrict operations, or require relinquishing rights to technologies/product candidates[278](index=278&type=chunk)[280](index=280&type=chunk) - The company faces intense competition from existing and future drugs marketed by large companies with substantially greater resources[283](index=283&type=chunk)[286](index=286&type=chunk) - Competition and technological change may render product candidates and technologies less attractive or obsolete, as other companies may develop products earlier, obtain approval faster, or develop more effective treatments[289](index=289&type=chunk)[290](index=290&type=chunk) [Risks Related to Our Intellectual Property](index=89&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Details risks concerning intellectual property, including licensing, patents, and trade secrets - The company is significantly dependent on its exclusive license with Minoryx Therapeutics S.L. for the SEE-Tx® platform; termination of this license would materially adversely affect the business[298](index=298&type=chunk)[299](index=299&type=chunk) - Ability to obtain and enforce patents is uncertain; patents may be challenged, invalidated, or circumvented, and may not provide significant protection against competitors[300](index=300&type=chunk)[304](index=304&type=chunk) - Reliance on trade secrets, confidentiality, and security measures may not adequately protect proprietary information, and others may independently develop similar technologies[308](index=308&type=chunk) - Involvement in intellectual property lawsuits (infringement, validity challenges) could be expensive, time-consuming, divert management attention, and result in significant liabilities or restrictions on product sales[310](index=310&type=chunk)[311](index=311&type=chunk)[316](index=316&type=chunk) [Risks Related to Third Parties and Collaborators](index=95&type=section&id=Risks%20Related%20to%20Third%20Parties%20and%20Collaborators) Explains risks associated with reliance on third-party contractors and collaboration agreements - Reliance on third parties (CROs, consultants) for preclinical studies and clinical trials means less control over timing and execution, with potential for delays, increased costs, or denial of regulatory approval if performance is unsatisfactory or non-compliant with GCPs[226](index=226&type=chunk)[321](index=321&type=chunk)[322](index=322&type=chunk) - Dependence on third-party CMOs for manufacturing product candidates for clinical trials and commercial sale poses risks of insufficient quantities, unacceptable costs, supply chain disruptions (e.g., COVID-19, geopolitical conflicts), and non-compliance with cGMPs, which could delay or prevent development and commercialization[327](index=327&type=chunk)[329](index=329&type=chunk)[332](index=332&type=chunk)[334](index=334&type=chunk) - Collaborations are crucial but carry risks of termination, partners changing focus, insufficient resources, or disputes over proprietary rights, potentially delaying or terminating development and commercialization programs[336](index=336&type=chunk)[343](index=343&type=chunk)[346](index=346&type=chunk) - Failure to establish internal marketing, sales, and distribution capabilities or successful third-party arrangements could limit market creation and product sales, impacting revenues[338](index=338&type=chunk)[341](index=341&type=chunk)[342](index=342&type=chunk) [General Risk Factors](index=103&type=section&id=General%20Risk%20Factors) Covers broad risks such as internal controls, macroeconomic conditions, personnel, and regulatory compliance - The company previously identified and remediated material weaknesses in internal control over financial reporting, but future failures could lead to misstatements and harm business[347](index=347&type=chunk)[348](index=348&type=chunk)[349](index=349&type=chunk) - Global and macroeconomic conditions (credit market volatility, bank failures, inflation, geopolitical conflicts) could adversely affect revenue, financial condition, and operating goals, potentially delaying or abandoning development plans[351](index=351&type=chunk)[352](index=352&type=chunk) - Future growth requires expanding operations, increasing headcount, and improving controls, which may be difficult to manage effectively[353](index=353&type=chunk)[354](index=354&type=chunk) - Dependence on key personnel and the ability to attract/retain qualified employees is critical; loss of senior management or inability to hire specialized staff could harm business[355](index=355&type=chunk)[356](index=356&type=chunk)[357](index=357&type=chunk) - Relationships with healthcare providers and payors are subject to federal and state healthcare fraud and abuse laws, false claims laws, and privacy laws (e.g., Anti-Kickback Statute, False Claims Act, HIPAA), with potential for substantial penalties for non-compliance[362](index=362&type=chunk)[364](index=364&type=chunk)[367](index=367&type=chunk) - Market acceptance and sales depend on adequate reimbursement from third-party payors; cost containment measures and healthcare legislative reforms (e.g., ACA, IRA) could limit coverage and reduce drug prices[369](index=369&type=chunk)[370](index=370&type=chunk)[371](index=371&type=chunk)[375](index=375&type=chunk) - International operations expose the company to risks like different regulatory requirements, reduced intellectual property protection, economic/political instability, and compliance with anti-corruption laws (e.g., FCPA)[379](index=379&type=chunk)[380](index=380&type=chunk)[384](index=384&type=chunk) - Product liability lawsuits related to clinical trials or commercialized products could result in substantial liabilities, even with insurance, and harm the business[385](index=385&type=chunk)[386](index=386&type=chunk) - Stringent and evolving data privacy and security laws (e.g., HIPAA, CCPA, GDPR) and cyber threats (ransomware, supply-chain attacks) pose risks of regulatory actions, litigation, fines, business disruptions, and reputational harm[387](index=387&type=chunk)[390](index=390&type=chunk)[391](index=391&type=chunk)[392](index=392&type=chunk)[397](index=397&type=chunk)[398](index=398&type=chunk)[399](index=399&type=chunk)[400](index=400&type=chunk)[405](index=405&type=chunk) [Risks Related to Ownership of Our Common Stock](index=124&type=section&id=Risks%20Related%20to%20Ownership%20of%20Our%20Common%20Stock) Discusses risks pertinent to common stock ownership, including price volatility and dilution - The market price for common stock has been and is likely to remain highly volatile, influenced by R&D results, financial estimates, competition, regulatory actions, and macroeconomic conditions[410](index=410&type=chunk)[413](index=413&type=chunk)[415](index=415&type=chunk) - Securities class action litigation is a risk for volatile stocks, potentially leading to substantial costs and diversion of management attention[413](index=413&type=chunk) - Failure to meet Nasdaq listing requirements could result in delisting, leading to limited market quotations, reduced trading activity, and decreased ability to raise future financing[414](index=414&type=chunk)[416](index=416&type=chunk) - Operating as a public company incurs increased costs and requires substantial management time for compliance initiatives and corporate governance[414](index=414&type=chunk) - The company does not anticipate paying dividends, so stockholders must rely on stock appreciation for returns, which is uncertain[424](index=424&type=chunk) - Sales of a substantial number of shares by existing stockholders or future equity raises could cause the stock price to fall and dilute existing ownership[425](index=425&type=chunk)[426](index=426&type=chunk) - Anti-takeover provisions in organizational documents and Delaware law might discourage or delay acquisition attempts[429](index=429&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=134&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) Reports no unregistered equity sales and details the use of proceeds from the March 2021 IPO [Sales of Unregistered Securities](index=134&type=section&id=Sales%20of%20Unregistered%20Securities) Confirms that no unregistered sales of equity securities occurred during the period - No unregistered sales of equity securities occurred[435](index=435&type=chunk) [Use of Proceeds from Initial Public Offering of Common Stock](index=134&type=section&id=Use%20of%20Proceeds%20from%20Initial%20Public%20Offering%20of%20Common%20Stock) Details the net proceeds from the IPO and confirms no material change in their planned use - The IPO, closed on **March 22, 2021**, generated net proceeds of **$40.5 million** from **4,181,818 common shares** at **$11.00 per share**[436](index=436&type=chunk) - There has been no material change in the planned use of proceeds from the IPO[437](index=437&type=chunk) [Item 3. Defaults Upon Senior Securities.](index=134&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities.) States that this item is not applicable to the company - Not applicable[438](index=438&type=chunk) [Item 4. Mine Safety Disclosures](index=134&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) States that this item is not applicable to the company - Not applicable[439](index=439&type=chunk) [Item 5. Other Information](index=134&type=section&id=Item%205.%20Other%20Information) States that this item is not applicable to the company - Not applicable[440](index=440&type=chunk) [Item 6. Exhibits](index=136&type=section&id=Item%206.%20Exhibits) Lists all exhibits filed with the Form 10-Q, including certifications and XBRL documents - The report includes certifications from the Principal Executive Officer and Principal Financial Officer (**Exhibits 31.1, 31.2, 32.1**)[442](index=442&type=chunk)[443](index=443&type=chunk) - Inline XBRL Instance Document and Taxonomy Extension Documents are filed as **Exhibits 101